Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.

Median chitotriosidase values showed highly significant commensurate reduction with eliglustat treatment in both cohorts ( P ≤ .005): 24% in non‐splenectomized switch patients and 38% in splenectomized switch patients after 2 years in this real‐world cohort compared to 63% after 4 years in ENCORE. 20 The safety of daily oral eliglustat cannot be assessed in the Registry, because adverse event data and reasons for discontinuing treatment are not captured.

also in 132,142 other papers